{"id":4971,"date":"2024-08-01T10:00:16","date_gmt":"2024-08-01T14:00:16","guid":{"rendered":"https:\/\/cytel.agencyukdev.com\/?post_type=perspectives&p=4971"},"modified":"2024-08-06T06:48:19","modified_gmt":"2024-08-06T10:48:19","slug":"the-role-of-external-data-in-oncology-drug-development","status":"publish","type":"perspectives","link":"https:\/\/cytel.agencyukdev.com\/fr\/perspectives\/the-role-of-external-data-in-oncology-drug-development\/","title":{"rendered":"The Role of External Data in Oncology Drug Development"},"content":{"rendered":"

Written by Natalia Muhlemann, Grace Hsu, and Evie Merinopoulou.\u00a0<\/em><\/p>\n

Randomized controlled trials (RCTs) remain the gold standard for <\/span>the <\/span>evaluation of <\/span>the safety and effectiveness of <\/span>a new treatment. However, in <\/span>a number of<\/span> cases alternative approaches <\/span>leveraging<\/span> external data<\/span> (i.e., data from outside of a clinical trial)<\/span> \u2014\u00a0<\/span>ranging from single arm trials to augmented RCT<\/span>s \u2014<\/span>\u00a0<\/span>can be <\/span>appropriate<\/span>. <\/span>Here<\/span>,<\/span> we discuss <\/span>how to <\/span>leverage<\/span> and incorporat<\/span>e<\/span> external data in<\/span> drug development<\/span>,<\/span> focusing on the<\/span> us<\/span>e of<\/span> external <\/span>control arms and Bayesian borrowing<\/span>.\u00a0<\/span>\u00a0<\/span><\/span>\u00a0<\/span><\/p>\n

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Use of external data in drug development<\/span><\/span>\u00a0<\/span><\/h2>\n

In early phases of drug development, single arm trials with external controls<\/a>\/comparisons can be used for efficacy signal-finding or dose selection to inform internal decision-making. Single arm trials with external control or augmented trials can be used in later phases for e<\/span>stablishing natural history of disease, isolation of treatment effect or supportive comparative efficacy.\u00a0<\/span>\u00a0<\/span><\/p>\n

For example, in rare diseases<\/a>, rare genetic subtypes of a disease, or in a debilitating and life threating disease, it may be not feasible or ethical to enroll the number of patients required for a conventional well-powered RCT. In the post-approval setting, studies with external controls can be used for comparative effectiveness to inform reimbursement decisions and clinical guidelines.\u00a0<\/span>\u00a0<\/span><\/p>\n

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Multi-step process to incorporate external data<\/span><\/span>\u00a0<\/span><\/h2>\n

Leveraging external data in drug development requires thoughtful evaluation and planning.\u00a0<\/span>\u00a0<\/span><\/p>\n

To begin, define the key objectives and assess the clinical rationale by asking the following questions:<\/span>\u00a0<\/span><\/p>\n